Date: 2017-02-16
Type of information: Initiation of the trial
phase: 1
Announcement: initiation of the trial
Company: Global Blood Therapeutics (USA - CA)
Product: GBT440
Action
mechanism: hemoglobin modifier. GBT440 is being developed as an oral, once-daily therapy for patients with sickle cell disease. GBT440 works by increasing hemoglobin's affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT believes GBT440 blocks polymerization and the resultant sickling of red blood cells (RBCs). With the potential to restore normal hemoglobin function and improve oxygen delivery, GBT440 may be capable of modifying the progression of sickle cell disease. The FDA has granted GBT440 both Fast Track and Orphan Drug designation for the treatment of patients with sickle cell disease in recognition of the critical need for new treatments.
Disease:
Therapeutic area: Hematological diseases
Country:
Trial
details: The open-label Basecamp study is evaluating the safety of GBT440 and its effect on oxygen saturation in fit healthy volunteers at rest and following maximal exercise under normoxic (normal levels of oxygen in the blood) and hypoxic conditions. Approximately 12 healthy volunteers, ages 18 to 50 years, will be enrolled and will receive 900 mg of GBT440 administered orally, once daily for 14 days. The primary endpoint of the study is the change in oxygen saturation at rest and during maximal exercise, measured under hypoxic conditions. Secondary endpoints include the change in VO2-max, cardiac output, dyspnea (difficult or labored breathing) – all measured under normoxic and hypoxic conditions at Day 15 compared with baseline and safety.
Latest
news: * On February 16, 2017, Global Blood Therapeutics announced initiation of the Phase 1 Basecamp (GBT440-011) clinical trial, which is evaluating the physiologic effects of GBT440 in fit healthy volunteers under hypoxic conditions and exercise conditions that maximally stress the function of the heart and lungs. Building on data from animal models showing improved oxygen delivery under hypoxic conditions, the Basecamp study is part of a clinical development program that is evaluating GBT440 as a potential treatment for hypoxemia and will complement the data from the ongoing phase 2 idiopathic pulmonary fibrosis studies.
